VRDN-003-303, Safety Study
Research type
Research Study
Full title
A randomized, double-masked, controlled, safety and tolerability study of VRDN-003 in participants with thyroid eye disease (TED)
IRAS ID
1011456
Contact name
N/A N/A
Contact email
Sponsor organisation
Viridian Therapeutics Inc.
Research summary
Thyroid eye disease (TED) is an autoimmune condition (a condition where the body attacks itself) most commonly related to the overproduction of thyroid hormones (hyperthyroidism). TED causes pain, redness and swelling in the tissues in and around the eyes, leading to bulging of the eyes (proptosis), double vision (diplopia), reduced movement of one or both eyes (restricted duction and version), and increased pressure on the nerves in the eyes, eventually leading to blindness.
Treatment has historically included vitamins, corticosteroids, topical agents, and antibiotics, though as symptoms become more severe, the only alternatives until recently have been surgery and radiation therapy. Biologic therapies have been used to treat TED, but there is little supportive clinical data, and none are licensed for the treatment of TED.
Teprotumumab, a fully human antibody (a laboratory-produced protein that acts like a human antibody) which targets IGF-1R (a protein present on the surface of eye cells) was recently approved for the treatment of TED in the USA and active TED in Brazil, the Kingdom of Saudia Arabia and Japan. In clinical trials, teprotumumab was more effective than placebo (a sham treatment with no active drug) in reducing TED symptoms, though is burdensome for participants, requiring 8 infusions over 24 weeks at an infusion centre.
VRDN-003, also a human antibody which targets IGF-1R, is thought to disrupt inflammation which contributes to TED, relieving symptoms. However, unlike teprotumumab, VRDN-003 is given as subcutaneous (SC) injections (injections given into the layer of fat between the skin and muscle), thus relieving the participants of the burden of infusions.
This study, sponsored by Viridian Therapeutics, Inc., will test if VRDN-003 is safe and tolerable in participants with TED when given as SC injections every 4 or 8 weeks, for a total of 6 injections. Approximately 284 participants are expected to take part across roughly 65 locations.
REC name
London - Westminster Research Ethics Committee
REC reference
25/LO/0175
Date of REC Opinion
7 Apr 2025
REC opinion
Further Information Favourable Opinion